Orphagen Pharmaceuticals, a privately-held emerging pharmaceutical company, announced today that a total of $622,971 in grants was awarded under the Qualifying Therapeutic Discovery Project (QTDP) program provided for in the Health Care and Education Reconciliation Act of 2010. The QTDP funding will support Orphagen’s technology and expertise in the discovery of innovative first-in-class drugs targeting orphan nuclear receptors.

“The QTDP grants recognize the potential of our drug discovery programs,” said Scott Thacher, CEO of Orphagen. “We received awards for all of the programs for which we submitted applications, and we can accelerate these programs as a result,” said Thacher.
Because Orphagen specializes in early stage, small molecule discovery, this funding will have a significant impact on new technology development in several of Orphagen’s programs:

  • improved treatments for dry age-related macular degeneration and retinitis pigmentosa.
  • treatment of adrenocortical cancer based on the orphan target steroidogenic factor-1 (SF-1), which is elevated in the tumor tissue of patients suffering from this devastating disease.
  • novel orphan receptor drug targets for cancer, diseases of the immune system and the eye.

Orphagen has made important discoveries in identifying potential drug ligands to previously unexplored orphan receptors, putative drug targets identified as part of the human genome project. Orphagen has captured a first-mover position for three of these targets.
Many global pharmaceutical companies view discovery research as too risky, and are increasingly relying on external sources of innovation. Orphagen holds a strong intellectual property position because of its first-to-ligand approach in several programs, and plans to pursue future drug development through partners with complementary capabilities in pre-clinical and clinical research.

About Orphagen: Orphagen discovers drug candidates for potential drug targets for which small molecule ligands–potential drug-like molecules–have yet to be identified. Its goal is to identify, characterize, and position a new class of drug so that pre-clinical and clinical development can be initiated with partners and/or outside sources of funding. These targets come from the nuclear receptor family of drug targets. On a per target basis, the nuclear receptors are one of the most successful target classes known to the pharmaceutical industry. Targets of interest to Orphagen encompass several of the so-called orphan nuclear receptors—potential therapeutic receptors that have yet to be exploited by the pharmaceutical industry.

For more information, contact: Scott Thacher, (858) 481-6191